NCT02828592 · Northside Hospital, Inc.
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
What this study is about
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding.
View original scientific description
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%.
Interventions
DRUG
Fludarabine
30 mg/m2 IV QD x 5 days (Days -6 to -2)
DRUG
Cyclophosphamide
14.5 mg/kg/day IV x 2 doses (Days -6 \& -5)
RADIATION
Total Body Irradiation
300 cGy x1 dose (Day -1)
DRUG
Rabbit ATG
1.5 mg/kg/day x 3 days (Days -3 to -1)
DRUG
Cyclophosphamide
Post-transplant: 50 mg/kg IV QD (Day +3 to +4)
Primary outcome measures
Demonstrate sustained engraftment after T-cell replete HLA-mismatched haploidentical bone marrow transplantation by collecting chimerism tests monthly following transplant
Time frame: 2 years
Hypothesis is that following preparative regimen and bone marrow transplantation, the 30-day graft failure rate will be \<30%.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
- Age \<= 65 years for previously treated and \<= 75 years for previously treated patients
- KPS \>= 70%
- Aplastic Anemia that meets the following criteria: Peripheral Blood (must fulfill 2 of 3):
- \<500 PMN/mm3
- \<20,000 platelets
- absolute reticulocyte count \<40,000/microL Bone Marrow (must be either):
- markedly hypocellular (\<25% of normal cellularity)
- moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above
Exclusion criteria
- poor cardiac function (LVEF \<40%)
- poor pulmonary function (FEV1 \& FVC \<50% predicted)
- poor liver function (bili \>= 2mg/dL)
- poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40mL/min)
- prior allogeneic transplant
Where
- Atlanta, Georgia
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 20, 2026 · Source of record for eligibility and locations